Orphan drug Arcalyst debuts for two rare inflammatory diseases


Rilonacept approved as orphan drug for Cryopyrin-Associated Periodic Syndromes (CAPS).

Orphan drug-designated rilonacept (approval from the Food & Drug Administration. The new therapy is indicated for the treatment of two Cryopyrin-Associated Periodic Syndrome (CAPS) disorders - Familial Cold Auto-Inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS), both chronic inflammatory diseases. Rilonacept inhibits the action of interleukin-1, a signaling protein that regulates inflammatory processes and is present in excess in both FCAS and MWS patients. The approval was based on a clinical study that utilized a patient questionnaire to rate five signs and symptoms of CAPS - joint pain, fever/chills, rash, eye redness/pain, and fatigue. Patients treated with rilonacept noted an onset of relief of symptoms within several days of initiating therapy and experienced an improvement in overall symptom scores compared with those receiving placebo that was sustained throughout treatment. The inherited disorders affect approximately 300 people in the United States and are associated with a gene mutation in the CIAS 1 gene. Regeneron said the new product will be launched within 30 days of approval.

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